Molecular Therapy-Methods & Clinical Development

Article	Citations
Airways Expression of SARS-CoV-2 Receptor, ACE2, and TMPRSS2 Is Lower in Children Than Adults and Increases with Smoking and COPD	221
Oncolytic Viruses and the Immune System: The Dynamic Duo	154
Barriers and Strategies of Cationic Liposomes for Cancer Gene Therapy	127
The Two Faces of ACE2: The Role of ACE2 Receptor and Its Polymorphisms in Hypertension and COVID-19	108
Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies	88
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors	85
CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain	78
Synthetic mRNA Encoding VEGF-A in Patients Undergoing Coronary Artery Bypass Grafting: Design of a Phase 2a Clinical Trial	77
Adeno-Associated Viral Vectors in Neuroscience Research	76
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning	74
Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease	67
Circular RNAs: Promising Molecular Biomarkers of Human Aging-Related Diseases via Functioning as an miRNA Sponge	66
Intramuscular Delivery of Replicon RNA Encoding ZIKV-117 Human Monoclonal Antibody Protects against Zika Virus Infection	64
CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice	63
Production of Lentiviral Vectors Using Suspension Cells Grown in Serum-free Media	62
Use of Nanovesicles from Orange Juice to Reverse Diet-Induced Gut Modifications in Diet-Induced Obese Mice	62
Immune Response Mechanisms against AAV Vectors in Animal Models	57
AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria	56
Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors	55
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?	54
Preclinical Toxicology of rQNestin34.5v.2: An Oncolytic Herpes Virus with Transcriptional Regulation of the ICP34.5 Neurovirulence Gene	53
Acerola exosome-like nanovesicles to systemically deliver nucleic acid medicine via oral administration	52
Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles	51
qPCR and qRT-PCR analysis: Regulatory points to consider when conducting biodistribution and vector shedding studies	51
Creation of a High-Yield AAV Vector Production Platform in Suspension Cells Using a Design-of-Experiment Approach	50

Increasing CRISPR Efficiency and Measuring Its Specificity in HSPCs Using a Clinically Relevant System	46
Superior Expansion and Cytotoxicity of Human Primary NK and CAR-NK Cells from Various Sources via Enriched Metabolic Pathways	43
Development of Large-Scale Downstream Processing for Lentiviral Vectors	43
Mechanistic model for production of recombinant adeno-associated virus via triple transfection of HEK293 cells	42
Enhancing Chimeric Antigen Receptor T Cell Anti-tumor Function through Advanced Media Design	42
piggyBac system to co-express NKG2D CAR and IL-15 to augment the in vivo persistence and anti-AML activity of human peripheral blood NK cells	42
High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models	41
AAV-S: A versatile capsid variant for transduction of mouse and primate inner ear	40
Enhanced expression of immune checkpoint receptors during SARS-CoV-2 viral infection	40
Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy	39
Development of a scalable and robust AEX method for enriched rAAV preparations in genome-containing VCs of serotypes 5, 6, 8, and 9	39
AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity	39
Modified mRNA-LNP Vaccines Confer Protection against Experimental DENV-2 Infection in Mice	38
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy	37
Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID	37
Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates	36
CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice	35
Anion-exchange HPLC assay for separation and quantification of empty and full capsids in multiple adeno-associated virus serotypes	35
Automated generation of gene-edited CAR T cells at clinical scale	35
Characterization of AAV-mediated dorsal root ganglionopathy	35
Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry	34
Characterization of AAV-Specific Affinity Ligands: Consequences for Vector Purification and Development Strategies	34
Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy	34
Overcoming Compensatory Mechanisms toward Chronic Drug Administration to Ensure Long-Term, Sustainable Beneficial Effects	33
iPSC-Derived Intestinal Organoids from Cystic Fibrosis Patients Acquire CFTR Activity upon TALEN-Mediated Repair of the p.F508del Mutation	33
Allele-Specific Prevention of Nonsense-Mediated Decay in Cystic Fibrosis Using Homology-Independent Genome Editing	33
A Single Intravenous Injection of AAV-PHP.B-hNDUFS4 Ameliorates the Phenotype of Ndufs4 Mice	33
Pharmacological interventions enhance virus-free generation of TRAC-replaced CAR T cells	32
The impact of lentiviral vector genome size and producer cell genomic to gag-pol mRNA ratios on packaging efficiency and titre	32
Empowering Retinal Gene Therapy with a Specific Promoter for Human Rod and Cone ON-Bipolar Cells	31
Pooled Screens Identify GPR108 and TM9SF2 as Host Cell Factors Critical for AAV Transduction	31
CERE-120 Prevents Irradiation-Induced Hypofunction and Restores Immune Homeostasis in Porcine Salivary Glands	30
Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals	30
A flexible, thermostable nanostructured lipid carrier platform for RNA vaccine delivery	30
Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2	30
Exosomes and organ-specific metastasis	30
Transplantation of miPSC/mESC-derived retinal ganglion cells into healthy and glaucomatous retinas	30
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations	30
AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction	29
Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients	29
Clinical Development of Gene Therapies: The First Three Decades and Counting	29
Rosuvastatin Enhances VSV-G Lentiviral Transduction of NK Cells via Upregulation of the Low-Density Lipoprotein Receptor	28
Optimized Assessment of qPCR-Based Vector Copy Numbers as a Safety Parameter for GMP-Grade CAR T Cells and Monitoring of Frequency in Patients	28
Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD	28
Enriching leukapheresis improves T cell activation and transduction efficiency during CAR T processing	27
Systemic and local immune responses to intraocular AAV vector administration in non-human primates	27
Assessment of genome packaging in AAVs using Orbitrap-based charge-detection mass spectrometry	27
Soluble Klotho Improves Hepatic Glucose and Lipid Homeostasis in Type 2 Diabetes	27
Purification of Human CD34+CD90+ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy	27
Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec	26

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease	26
Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease	26
In vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system	26
Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries	26
Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration	26
Rapid Lentiviral Vector Producer Cell Line Generation Using a Single DNA Construct	26
Optimization of 5′ Untranslated Region of Modified mRNA for Use in Cardiac or Hepatic Ischemic Injury	26
Hair Cell Transduction Efficiency of Single- and Dual-AAV Serotypes in Adult Murine Cochleae	26
Human Mesenchymal Stem Cell Secretome Exhibits a Neuroprotective Effect over In Vitro Retinal Photoreceptor Degeneration	26
Evaluating the Potential of T Cell Receptor Repertoires in Predicting the Prognosis of Resectable Non-Small Cell Lung Cancers	25
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors	25
Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper	25
Simple and Fast SEC-Based Protocol to Isolate Human Plasma-Derived Extracellular Vesicles for Transcriptional Research	25
Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease	25
Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II	25
Factors Impacting Efficacy of AAV-Mediated CRISPR-Based Genome Editing for Treatment of Choroidal Neovascularization	24
iMATCH: an integrated modular assembly system for therapeutic combination high-capacity adenovirus gene therapy	24
Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease	24
Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter	24
rAAV2-Retro Enables Extensive and High-Efficient Transduction of Lower Motor Neurons following Intramuscular Injection	23
Metformin, an AMPK Activator, Inhibits Activation of FLSs but Promotes HAPLN1 Secretion	23
Cell therapy with hiPSC-derived RPE cells and RPCs prevents visual function loss in a rat model of retinal degeneration	23
Characteristics of the Coronavirus Disease 2019 and related Therapeutic Options	23
In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency	23
High-Resolution Histological Landscape of AAV DNA Distribution in Cellular Compartments and Tissues following Local and Systemic Injection	23
Comparison of AAV-Mediated Optogenetic Vision Restoration between Retinal Ganglion Cell Expression and ON Bipolar Cell Targeting	22
In Situ Detection of Adeno-associated Viral Vector Genomes with SABER-FISH	22
Promoter usage regulating the surface density of CAR molecules may modulate the kinetics of CAR-T cells in vivo	22
Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries	22
Current landscape of clinical development and approval of advanced therapies	22
Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application	22
CAR T Cell Generation by piggyBac Transposition from Linear Doggybone DNA Vectors Requires Transposon DNA-Flanking Regions	22
Monolayer platform using human biopsy-derived duodenal organoids for pharmaceutical research	22
AAV6 Vexosomes Mediate Robust Suicide Gene Delivery in a Murine Model of Hepatocellular Carcinoma	22
Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses	22
A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation	22
Chemically Defined, High-Density Insect Cell-Based Expression System for Scalable AAV Vector Production	22
Lentiviral Vector Production Titer Is Not Limited in HEK293T by Induced Intracellular Innate Immunity	22
CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich’s Ataxia	22
Nanoplasmid Vectors Co-expressing Innate Immune Agonists Enhance DNA Vaccines for Venezuelan Equine Encephalitis Virus and Ebola Virus	21
Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations	21
Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates	21
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys	21
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8	21
Inhibition of KDM1A activity restores adult neurogenesis and improves hippocampal memory in a mouse model of Kabuki syndrome	21
Cerebral Organoids: A Human Model for AAV Capsid Selection and Therapeutic Transgene Efficacy in the Brain	21
Engineered extracellular vesicles directed to the spike protein inhibit SARS-CoV-2	21
Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors	21
CRISPAltRations: A validated cloud-based approach for interrogation of double-strand break repair mediated by CRISPR genome editing	20
Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering	20
A Single “All-in-One” Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair	20
Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice	20
ISPD Overexpression Enhances Ribitol-Induced Glycosylation of α-Dystroglycan in Dystrophic FKRP Mutant Mice	20
Circulating miR-4763-3p Is a Novel Potential Biomarker Candidate for Human Adult Fulminant Myocarditis	20
A Long Intergenic Non-coding RNA, LINC01426, Promotes Cancer Progression via AZGP1 and Predicts Poor Prognosis in Patients with LUAD	20
Effective Multi-lineage Engraftment in a Mouse Model of Fanconi Anemia Using Non-genotoxic Antibody-Based Conditioning	20
Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss	20
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping	20
Applying a clinical lens to animal models of CAR-T cell therapies	20
Development of AAV Variants with Human Hepatocyte Tropism and Neutralizing Antibody Escape Capacity	20
Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity	20
Antigen modifications improve nucleoside-modified mRNA-based influenza virus vaccines in mice	20
Structural basis for the neurotropic AAV9 and the engineered AAVPHP.eB recognition with cellular receptors	20
A Rapid and Sensitive Nucleic Acid Amplification Technique for Mycoplasma Screening of Cell Therapy Products	20
Improved diabetic wound healing by LFcinB is associated with relevant changes in the skin immune response and microbiota	20
Systematic improvements in lentiviral transduction of primary human natural killer cells undergoing ex vivo expansion	19
Gene therapy strategies for idiopathic pulmonary fibrosis: recent advances, current challenges, and future directions	19
Non-toxic HSC Transplantation-Based Macrophage/Microglia-Mediated GDNF Delivery for Parkinson’s Disease	19
Circulating neurofilament light chain as a promising biomarker of AAV-induced dorsal root ganglia toxicity in nonclinical toxicology species	19
Reversing Acute Kidney Injury Using Pulsed Focused Ultrasound and MSC Therapy: A Role for HSP-Mediated PI3K/AKT Signaling	19
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia	19
Lentiviral Vector Production from a Stable Packaging Cell Line Using a Packed Bed Bioreactor	19
CRISPR-Cpf1 Activation of Endogenous BMP4 Gene for Osteogenic Differentiation of Umbilical-Cord-Derived Mesenchymal Stem Cells	19
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells	19
Molecular subtyping and functional validation of TTK, TPX2, UBE2C, and LRP8 in sensitivity of TNBC to paclitaxel	19
Rapid Delivery of Nanobodies/VHHs into Living Cells via Expressing In Vitro-Transcribed mRNA	19
Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice	19
A qPCR Method for AAV Genome Titer with ddPCR-Level of Accuracy and Precision	19
AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains	19
In Vitro and In Vivo Amenability to Migalastat in Fabry Disease	19

Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors	19
Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series	18
Enhanced Inner-Ear Organoid Formation from Mouse Embryonic Stem Cells by Photobiomodulation	18
Wound Healing Properties of Histatin-5 and Identification of a Functional Domain Required for Histatin-5-Induced Cell Migration	18
Depletion of high-content CD14+ cells from apheresis products is critical for successful transduction and expansion of CAR T cells during large-scale cGMP manufacturing	18
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse	18
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina	18
A hierarchical and collaborative BRD4/CEBPD partnership governs vascular smooth muscle cell inflammation	18
Optimization of AAV6 transduction enhances site-specific genome editing of primary human lymphocytes	18
Intralingual and Intrapleural AAV Gene Therapy Prolongs Survival in a SOD1 ALS Mouse Model	17
Comprehensive and systemic optimization for improving the yield of SARS-CoV-2 spike pseudotyped virus	17
Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine Model	17
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal	17
Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia	17
High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone	17
Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation	17
Development and Optimization of a Hydrophobic Interaction Chromatography-Based Method of AAV Harvest, Capture, and Recovery	17
Treatment of Hypertensive Heart Disease by Targeting Smad3 Signaling in Mice	17
Novel Non-integrating DNA Nano-S/MAR Vectors Restore Gene Function in Isogenic Patient-Derived Pancreatic Tumor Models	17
Skeletal Muscle Is an Antigen Reservoir in Integrase-Defective Lentiviral Vector-Induced Long-Term Immunity	17
A pro-inflammatory mediator USP11 enhances the stability of p53 and inhibits KLF2 in intracerebral hemorrhage	17
Autologous antigen-presenting cells efficiently expand piggyBac transposon CAR-T cells with predominant memory phenotype	16
Generation of Nonhuman Primate Model of Cone Dysfunction through In Situ AAV-Mediated CNGB3 Ablation	16
Analysis of Aflibercept Expression in NHPs following Intravitreal Administration of ADVM-022, a Potential Gene Therapy for nAMD	16
Directed Evolution of AAV Serotype 5 for Increased Hepatocyte Transduction and Retained Low Humoral Seroreactivity	16
Spatiotemporal in vivo tracking of polyclonal human regulatory T cells (Tregs) reveals a role for innate immune cells in Treg transplant recruitment	16
Manufacturing NKG2D CAR-T cells with piggyBac transposon vectors and K562 artificial antigen-presenting cells	16
Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors	16
Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH	16
A third dose of the unmodified COVID-19 mRNA vaccine CVnCoV enhances quality and quantity of immune responses	16
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers	16
Analysis of thermally driven structural changes, genome release, disassembly, and aggregation of recombinant AAV by CDMS	16
Discovery of key genes as novel biomarkers specifically associated with HPV-negative cervical cancer	16
7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy	16
C3 Transferase-Expressing scAAV2 Transduces Ocular Anterior Segment Tissues and Lowers Intraocular Pressure in Mouse and Monkey	16
A DNA Vaccine That Encodes an Antigen-Presenting Cell-Specific Heterodimeric Protein Protects against Cancer and Influenza	15
Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration	15
rAAV8 and rAAV9-Mediated Long-Term Muscle Transduction with Tacrolimus (FK506) in Non-Human Primates	15
Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy	15
MTA1, a Target of Resveratrol, Promotes Epithelial-Mesenchymal Transition of Endometriosis via ZEB2	15
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia	15
Novel GAA Variants and Mosaicism in Pompe Disease Identified by Extended Analyses of Patients with an Incomplete DNA Diagnosis	15
Vector Copy Distribution at a Single-Cell Level Enhances Analytical Characterization of Gene-Modified Cell Therapies	15
Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice	15
DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis	15
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography	15
Visceral adipose tissue-directed FGF21 gene therapy improves metabolic and immune health in BTBR mice	15
Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS	15
Lentiviral and adeno-associated vectors efficiently transduce mouse T lymphocytes when targeted to murine CD8	15
Analytical band centrifugation for the separation and quantification of empty and full AAV particles	15
Efficient In Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65	15
Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method	15
Co-transducing B7H3 CAR-NK cells with the DNR preserves their cytolytic function against GBM in the presence of exogenous TGF-β	14
Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina	14
Differential T cell immune responses to deamidated adeno-associated virus vector	14
UTX/KDM6A deletion promotes the recovery of spinal cord injury by epigenetically triggering intrinsic neural regeneration	14
Targeted knockdown of the adenosine A2A receptor by lipid NPs rescues the chemotaxis of head and neck cancer memory T cells	14
The Art and Science of Selecting a CD123-Specific Chimeric Antigen Receptor for Clinical Testing	14
GJB2 gene therapy and conditional deletion reveal developmental stage-dependent effects on inner ear structure and function	14
AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model	14
Expression of Neprilysin in Skeletal Muscle by Ultrasound-Mediated Gene Transfer (Sonoporation) Reduces Amyloid Burden for AD	14
Circulating Biomarkers in Muscular Dystrophies: Disease and Therapy Monitoring	14
Single AAV-Mediated CRISPR-SaCas9 Inhibits HSV-1 Replication by Editing ICP4 in Trigeminal Ganglion Neurons	14
Chronic 2-Fold Elevation of Endogenous GDNF Levels Is Safe and Enhances Motor and Dopaminergic Function in Aged Mice	14
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	14
Lipid nanoparticle mRNA systems containing high levels of sphingomyelin engender higher protein expression in hepatic and extra-hepatic tissues	14
Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy	14
Human-specific GAPDH qRT-PCR is an accurate and sensitive method of xenograft metastasis quantification	14
CRISPR-Mediated Base Conversion Allows Discriminatory Depletion of Endogenous T Cell Receptors for Enhanced Synthetic Immunity	14
Establishment of SLC15A1/PEPT1-Knockout Human-Induced Pluripotent Stem Cell Line for Intestinal Drug Absorption Studies	14
Design and Testing of Vector-Producing HEK293T Cells Bearing a Genomic Deletion of the SV40 T Antigen Coding Region	14
Decrease in Angiotensin-Converting Enzyme activity but not concentration in plasma/lungs in COVID-19 patients offers clues for diagnosis/treatment	14
Reproducible immortalization of erythroblasts from multiple stem cell sources provides approach for sustainable RBC therapeutics	14
Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs	14
Clinically relevant T cell expansion media activate distinct metabolic programs uncoupled from cellular function	14
Hypoxia-directed tumor targeting of CRISPR-Cas9 and HSV-TK suicide gene therapy using lipid nanoparticles	14
CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease	14
Accelerating clinical-scale production of BCMA CAR T cells with defined maturation stages	14